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December 7, 2009

Homing in on answers related to optimal combinations for induction therapy and the role of maintenance, Anne Quinn Young, MPH

Yesterday, more than 6,000 attendees packed into a hall the size of a small airport to hear Dr. Maria-Victoria Mateos from Dr. Jesus San Miguel’s group in Salamanca, Spain, deliver her plenary presentation at the ASH meeting. Just six abstracts out of thousands submitted for consideration are selected for plenary presentations at the meeting, so the myeloma community was thrilled that one of the six this year focused on our disease. The trial she presented built on the knowledge from the VISTA trial, which showed the overwhelming superiority of Velcade plus melphalan-prednisone versus melphalan-prednisone. The objective was to determine whether the best partner for Velcade was an IMiD (in this case, Thalomid – the only IMiD available in Spain when the study began) or an alkylator (in this case, melphalan) and whether there was a benefit to maintenance therapy. The results indicated that both combinations (VMP and VTP) had high response rates, include a high complete response (CR) rate, but VMP had fewer side effects. Furthermore, maintenance therapy – further bolstered the already high CR and near CR rates. Mateos also looked at using weekly Velcade which yielded high response rates while cutting the rate of peripheral neuropathy in half.

Following Mateos’s presentation were a series of abstracts looking at different induction regimens prior to high dose chemotherapy and stem cell transplant. For the most part, the results were preliminary as the trials were either phase I/II trials or had not reached endpoints like median time to progression or overall survival. Though the data are preliminary, several themes emerged though in all of these cases, more data are needed:

  1. Maintenance therapy appears to enhance the depth of response
  2. Weekly Velcade may provide similar benefit but with a lower rate of neuropathy
  3. It is not yet clear whether 3 or 4 drugs need to be combined upfront to maximize outcomes – data are not yet mature enough to show the benefits outweigh the added toxicities
  4. Data continue to indicate that Velcade overcomes adverse outcomes previously associated with certain genetic abnormalities such as the translocation of chromosomes 4 and 14

Finally, it is critical to note that none of the larger European trials included Revlimid as it was not available when these trials began several years ago so the regimens tested are not indicative of current standards of care in the US. We are fortunate that treatment options continue to evolve but this means that at times, when large phase III trials are finally completed, one or more of the treatment regimens tested may no longer represent a reasonable standard of care.

The take home message is that while we don’t yet know the “best” regimen for untreated myeloma patients – whether they are transplant candidates or not – there are a number of important ongoing trials that will help us to answer this question. In the meantime, if you’re eligible, consider enrolling in a clinical trial to speed the pace of these trials for the benefit of the entire community.

December 6, 2009

MMRF and MMRC Report from ASH, Susan Kelley, MD

Representatives from the MMRF and MMRC are in New Orleans attending the 51st Annual Meeting of the American Society of Hematology, which runs from Friday Dec. 4 through Tuesday Dec. 8. This meeting is the leading worldwide gathering of experts and researchers in hematologic (blood) disorders. There is an increasing amount of preclinical and clinical research activity in myeloma reported at these meetings each year. This year the conference lists approximately 1100 abstracts published related to Myeloma laboratory research or Myeloma clinical observations and trials, including one of the major plenary session presentations on Sunday. On Friday, the MMRC convened a face-to–face meeting with the investigative members of the MMRC Project Review Committee, which brought together representatives from all of our member sites. The agenda included discussions of the MMRF/C strategic directions (business plan) and review of two potential new early clinical development drug candidates for possible clinical trials within the Consortium. Friday evening, the MMRF partnered with a CME provider to host a well-attended session entitled, “Evolving Therapeutic Concepts in Multiple Myeloma”. More than 700 ASH meeting attendees and patients from the local New Orleans area attended. Five esteemed Myeloma experts presented summary overviews and updates on the state of the art treatment issues.

On Saturday, members of the MMRF and MMRC participated in small group meetings with 10 pharma/biotech companies to discuss possible laboratory and clinical trial collaborations with candidate drugs of interest for MM. Among the initiatives discussed with the companies, was the possibility of conducting phase I-II trials of these new agents in combination with some of the backbone therapy that exists today. Each of the companies expressed interest in learning more about the collaborative trial support systems for conduct of Investigator Sponsored Clinical Trials within the Consortium, which we are establishing for kick off later this quarter.

Stay tuned to the MMRF Website for further updates on the data and highlights of presentations from ASH throughout the weekend.

December 3, 2009

FasterCures - Partnering for Cures, Susan Kelley, MD

Nearly 600 medical research executives, scientists, policy makers, and funders today convened in NY to explore novel development approaches and innovative funding strategies at the inaugural Partnering for Cures meeting. The MMRF and MMRC were well –represented at the meeting.

FasterCures, part of the Milken Institute, convened Partnering for Cures to facilitate multi-sector collaborations needed to turn a scientific discovery into an accessible therapy. The meeting was intended to highlight best practices in the conduct and management of medical research and approaches that could streamline the process. The leadership of the MMRF and MMRC was invited to make several presentations about the innovative collaborative research and development model that is the hallmark of the MMRF. Kathy Giusti, MMRF Founder and CEO, presented her perspectives as the leader of the MMRF during the opening plenary session on Wednesday morning. On that same panel was Bob Beall, CFF President and CEO and MMRF Board member. Bob's leadership in the area of venture philanthropy is unparalleled and we are incredibly lucky to have him as a member of our Board. Leading the panel was Margaret Anderson, Executive Director of FasterCures. Kudos to Margaret for her work on this amazing meeting and Mike Milken, head of Fastercures, for his insights into improving medical research.

Susan Kelley, MD, CMO of the MMRC, participated in a panel discussion to allow the audience to learn first-hand from “cure entrepreneurs”, nonprofit leaders who are implementing unique business models to produce dramatic results for patients. Louise Perkins, PhD, CSO of the MMRF, presented further details of the MMRF and MMRC collaborative model for myeloma drug research and development at a session which included many interested philanthropists, executives and scientists from various organizations. One-on-one connections between the meeting participants were facilitated through a number of partnering sessions.

Overall, it is invigorating to see a groundswell of interest to build bridges among the key stakeholders (patients, researchers and industry) to advance medical science in an approach we have pursued for many years.

November 24, 2009

The Changing Nature of Multiple Myeloma Research

Over the last few weeks, I have been privileged to participate in several scientific conferences on the subject of cancer research. The first of these meetings was held by the National Cancer Institute (NCI) in Virginia, the second was an international meeting in Boston on Pharmaceutical Oncology R&D and most recently another meeting was held in the same locale that is known as the Molecular Targets meeting sponsored by the American Association for Cancer Research (AACR), NCI and the European Oncology Research and Treatment Organization (EORTC). As a field, the nature of research is changing to embrace more applied, so-called translational, research. The NCI is taking a leadership position to push this transition along. Discoveries are being made at the bench and translated to the patient's bedside (and back again) at an ever increasing pace and laying the groundwork for personalized medicine in the future. What was most exciting at these meetings was hearing from international leaders and researchers that the MMRF and MMRC are widely recognized as leaders promoting continued advancements in myeloma research and drug development. Because of the tremendous collaborations that have been established involving researchers around the world, the stage is set for the next generation of treatments and for the coming wave of matching patients with drugs through personalized medical approaches.

From a scientific perspective, one of the most exciting discoveries described at the Molecular Targets meeting was around a gene called IDH1 which appears to be mutated in some types of cancers and is correspondingly subverted to a different purpose in tumor cells. These observations suggest that there is an entirely new collection of targets for drug intervention that could result in drugs specifically targeting the tumor and sparing the patient's normal cells and thereby exerting fewer side-effects than traditional chemotherapeutic approaches. Because of the work that the MMRF has already undertaken through the establishment of the MMRC Tissue Bank and through our successful sequencing of the myeloma tumor genome in many multiple myeloma patients' samples, multiple myeloma patients as a group stand to be among the first to benefit from these findings in the very near future.

-Louise M Perkins, PhD
 Chief Scientific Officer

October 5, 2009

Our Thoughts Are With Kenny Perry, Multiple Myeloma Research Foundation (MMRF)

We were so sorry to learn that Mildred Perry, the mother of golf pro Kenny Perry, passed away following a long battle with multiple myeloma. It is families like the Perry’s who inspire us to work urgently to bring patients better, more effective treatments, and we are truly saddened by their loss. Our thoughts are with Kenny and his family and friends during this difficult time, and we wish him the very best in next week’s President’s Cup.

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